Cost-Effectiveness Analysis of Pembrolizumab for BCG-Unresponsive Carcinoma in Situ of the Bladder - Expert Commentary

Pembrolizumab is approved to treat patients with BCG unresponsive, high-risk, non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy based on the results of the KEYNOTE-057 trial. However, pembrolizumab costs over $100,000 per year and has a significant rate of adverse effects. Compared to other treatment options, it is unknown if pembrolizumab is a cost-effective option in this setting.


A recent study by Wymer et al., published in the Journal of Urology, conducted a cost-effectiveness analysis of pembrolizumab compared to RCx or salvage intravesical chemotherapy (SIC). Two situations were investigated, the first for patients who are candidates for radical cystectomy (RCx), the second for those who are unwilling or unable to undergo RCx. The authors used decision-analytic Markov modeling of treatment options. Incremental Cost-Effectiveness Ratios (ICERs) were compared using a willingness-to-pay threshold of $100,000/Quality-adjusted life year (QALY). Up to 5-year time horizons were used.

The study revealed that pembrolizumab was not cost-effective relative to RCx or SIC in either situation. Interestingly, a more than 90% price reduction was needed to make pembrolizumab cost-effective compared to RCx or SIC. The investigators used several iterations in their calculations. Still, pembrolizumab was not cost-effective, even with a willingness-to-pay threshold of $500,000.

Moreover, the study showed that SIC would be only cost-effective relative to RCx if the cost of RCx increased over $13,228 compared with a baseline estimate of $11,527. The SIC would also be cost-effective relative to RCx if the 2-year risk of recurrence or metastasis were less than 55% or 5.9%, respectively. In these models, the authors used gemcitabine-docetaxel as the model for SIC.

Long-term and real-world data from the use of pembrolizumab in this setting may change the cost-effectiveness outcomes from the current models.

Written by: Bishoy M. Faltas, MD, Director of Bladder Cancer Research, Englander Institute for Precision Medicine, Weill Cornell Medicine, New York City, New York

Reference:

  1. Wymer KM, Sharma V, Saigal CS, Chamie K, Litwin MS, Packiam VT, et al. Cost-Effectiveness Analysis of Pembrolizumab for BCG-Unresponsive Carcinoma in Situ of the Bladder. J Urol. 2020 Dec 21;doi.10.1097/JU.0000000000001515. PMID: 33347775.

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